UniQure Halts AMT-191 Mid/High Doses for Fabry Disease After Liver Toxicities; Class Action Filed
UniQure’s Phase I/IIa AMT-191 trial showed all 11 Fabry patients achieved above-normal enzyme activity at three doses, with six stopping ERT and stable lyso-Gb3. High and mid dosing paused after liver enzyme toxicities and uniQure faces a shareholder suit over pivotal study design, with filings due April 13, 2026.
1. Positive Phase I/IIa Trial Results
UniQure’s AMT-191 gene therapy for Fabry disease demonstrated sustained above-normal α-Gal A enzyme activity in all 11 patients across low, mid and high dose cohorts. Six participants ceased bi-weekly ERT, maintaining stable lyso-Gb3 levels, with the longest-treated patient retaining efficacy for over one year.
2. Dosing Pause Over Safety Findings
Recruitment for mid and high dose groups has been paused following two dose-limiting liver enzyme elevations in the mid cohort, both resolving with steroid intervention. Previously reported serious events in the high-dose cohort include chest pain and stroke, though causality varied and remains under review.
3. Shareholder Class Action Details
A shareholder class action filed on behalf of investors who bought shares between September 24 and October 31, 2025 alleges that uniQure misrepresented its pivotal study design and downplayed potential BLA timeline delays. Investors must file lead plaintiff motions by April 13, 2026.